TREASURE topline results show evidence of
therapeutic impact
Study preliminary results analyses indicate
good potential for favorable results in Athersys’ MASTERS-2
study
Conference call today at 8:30 a.m.
ET
Athersys, Inc. (Nasdaq: ATHX) announced today that its partner,
HEALIOS K.K. (Healios), has reported topline results for its Japan
ischemic stroke study, TREASURE. The randomized, double-blind
placebo-controlled study included patients with moderate to
moderate-severe ischemic stroke (baseline NIHSS 8-20) with
administration of a single dose of MultiStem® cells (invimestrocel)
or placebo by intravenous infusion within 18-36 hours of the event.
The trial enrolled 206 patients and was conducted by 48 sites in
Japan.
Topline Results
- Improvement in pre-specified measures of functional
“independence” and good outcomes, such as mRS ≤2, Barthel Index ≥95
and Global Recovery, associated with MultiStem treatment.
- The primary endpoint, Excellent Outcome at 90 days, did not
reach statistical significance in this population.
- Overall, consistent improvement in essentially all measured
functional outcomes over time through one year, supporting
long-term impact on and continued improvement in the quality of
life of treated patients.
- High potential for success on Athersys’ MASTERS-2 primary
outcome measure, mRS shift, suggested by the results for the
TREASURE patients who were representative of the current enrollment
for MASTERS-2.
- No material differences in safety outcomes, including mortality
and life-threatening adverse events between the treatment and
placebo groups.
One Year
MultiStem
Placebo
*
Global Recovery
27.9%
15.7%
p<0.05
Barthel Index ≥95
35.6%
22.5%
p=0.05
Excellent Outcome
15.4%
10.8%
n.s.
Note: Excellent Outcome (mRS ≤1, NIHSS ≤1
and Barthel Index ≥95); Global Recovery (mRS ≤2, NIHSS change ≥75%
and Barthel Index ≥95). *Covariance adjustment based on
stratification factors in SAP.
“We are enthusiastic about the topline results from the TREASURE
ischemic stroke trial in Japan,” remarked Dan Camardo, Chief
Executive Officer of Athersys, Inc. “The preliminary data analyses
suggest a path forward for our partner, Healios, in Japan, and
gives us further confidence in our MASTERS-2 Phase 3 ischemic
stroke study being conducted in the United States, Europe and the
Asia-Pacific region. We look forward to continued collaboration
with Healios in working to bring this important therapy to the
market in Japan.”
Distinctive Characteristics of TREASURE Patient Population
and Favorable Read-through to MASTERS-2 Study
The TREASURE patient population was substantially older than the
MASTERS-1 population (and older than expected in the design of the
TREASURE study) with moderately greater stroke severity. The median
age for TREASURE patients was 78 years, compared to 63 years in the
MASTERS-1 study. The MASTERS-2 population is expected to be
significantly younger, with lower average stroke severity, than the
TREASURE population, based on current enrollment information.
Compared to the MASTERS-1 population, slower and less robust
recovery would be expected within the TREASURE patient group,
making measures of independence and good outcomes, such as mRS ≤2
and Barthel Index ≥95, more clinically relevant than “excellent
outcomes” or “ideal” health status post-stroke. The modified Rankin
Score (mRS) measures global disability on a 7-point scale and the
Barthel Index evaluates a patient’s ability to perform specific
activities of daily living on a 100-point scale, and both are
commonly used to assess clinical outcomes over time following
stroke.
The TREASURE study adds considerable new information about the
MultiStem treatment effect in older patients, aged 80 years and
older, not previously studied in MASTERS-1 and who are expected to
represent a limited proportion of subjects in the ongoing MASTERS-2
study. At the same time, it provides important information for
confirming and refining expectations for the MASTERS-2 study.
The results below, from the TREASURE study patients with the
most relevance for the MASTERS-2 study, suggest that MASTERS-2
would be expected to have a high probability of achieving success
on the primary endpoint, especially when taking into account the
larger MASTERS-2 sample size. Moreover, this does not account for
the expected positive contribution to the results from younger
patients in MASTERS-2, when compared to the representative TREASURE
sample described below.
TREASURE patient population relevant to
MASTERS-2*
Timepoint
MultiStem
Placebo
mRS Shift
(MASTERS-2 primary endpoint)
90 days
OR=1.7, p=0.13
365 days
OR=1.8, p=0.06
mRS ≤2
(key secondary endpoint)
90 days
46.4%
27.9%
p<0.05
365 days
50.0%
32.8%
p=0.06
* Representative TREASURE population
(patients <80 years, n=117). Covariance adjustment using
stratification factors, in accordance with MASTERS-2 SAP.
“The TREASURE results demonstrate therapeutic impact on multiple
clinically relevant endpoints in this population of older stroke
patients, with continued improvement over time,” commented Dr. John
Harrington, Chief Scientific Officer of Athersys, Inc. “We are
particularly pleased by the results for the younger TREASURE study
patients who are most relevant to our ongoing MASTERS-2 trial,
which we believe suggest a high potential for success in the
study.”
“The benefits provided to the TREASURE patients by MultiStem
treatment represent good recovery, are clinically meaningful and
should result in substantial improvement in quality of life for
many stroke patients,” commented David C. Hess, MD, Dean and
Presidential Distinguished Chair of Neurology at the Medical
College of Georgia at Augusta University, investigator and lead
author of the MASTERS-1 trial. “The TREASURE study enrolled an
elderly stroke population with a median age of 78 years, making
these results even more remarkable. We are excited about the
MASTERS-2 study and the potential to add a new, clinically
meaningful treatment option to stroke clinicians and patients.”
The Athersys and Healios teams plan to continue to analyze the
TREASURE results as additional data becomes available, including
the impact on biomarkers and more detailed evaluation of important
factors associated with the treatment effect, among other things.
Furthermore, Athersys plans to support regulatory engagement,
including together with Healios in Japan, to advance the ischemic
stroke program forward on its regulatory path. With respect to the
MASTERS-2 study, Athersys intends to continue its proactive efforts
to improve enrollment through site-expansion and site-productivity
initiatives to complete enrollment as soon as possible.
Key TREASURE Patient Characteristics
Overall, there was good balance between MultiStem and placebo
patient groups in age, severity and prior reperfusion treatment as
evident below.
MultiStem
Placebo
n
104
102
Age, median
79
78
Sex (male)
53.8%
54.9%
Severity
NIHSS, median
14.0
14.0
Reperfusion Status
No Prior
46.2%
49.0%
Prior (tPA or MR)
53.8%
51.0%
Other Studies Mentioned
MASTERS-1: completed 126-patient Phase 2 study of MultiStem
treatment for ischemic stroke, conducted by Athersys in the United
States and Europe.
MASTERS-2: ongoing 300-patient Phase 3 clinical trial of
MultiStem treatment for ischemic stroke, conducted by Athersys in
the United States (under Special Protocol Assessment), Europe and
Asia-Pacific. The program has Fast Track and RMAT designations.
Please refer to the following Healios disclosures for more
information -
https://ssl4.eir-parts.net/doc/4593/tdnet/2128690/00.pdf
Conference Call
Management will host a conference call today at 8:30 a.m. ET to
discuss the trial results. Participants may listen by viewing the
webcast online or may listen using the phone. We encourage
listeners to access the call using the webcast link to view slides
that will be discussed during the presentation. If you would like
to dial in using the phone, please register for the conference call
ahead of time using the phone registration link below. Once
registered, you will receive the toll-free number and a unique
conference ID.
Choose one method below:
Date
May 20, 2022
Time
8:30 a.m. (Eastern Time)
Live webcast registration
https://events.q4inc.com/attendee/903528413
Phone registration
https://conferencingportals.com/event/YqVRZKvZ
A replay of the event will be available at www.athersys.com
under the investors' section soon after the call has ended.
Investors may also call in for on-demand listening approximately
two hours after the completion of the call until 11:59 p.m. Eastern
Time on May 27, 2022, by dialing (800) 770-2030 or (647) 362-9199
and entering the access code 70781.
About Ischemic Stroke
Stroke represents an area where the clinical need is
particularly significant, since it is a leading cause of death and
serious disability worldwide, with a substantially impaired quality
of life for many stroke victims. Currently, there are nearly 17
million people who suffer a stroke globally and more than two
million stroke victims each year in the United States, Europe and
Japan, combined. Ischemic strokes, which represent the most common
form of stroke, are caused by a blockage of blood flow in the brain
that cuts off the supply of oxygen and nutrients and can result in
long-term or permanent disability due to neurological damage.
Unfortunately, current therapeutic options for ischemic stroke
victims are limited, since the only available treatments,
administration of the clot dissolving agent tPA, or “thrombolytic,”
or surgical intervention to remove the clot, must be conducted
within several hours of the occurrence of the stroke. As a
consequence of this limited time window, only a small percentage of
stroke victims are treated with the currently available
therapy—most simply receive supportive or “palliative” care. The
long-term costs of stroke are substantial, with many patients
requiring extended hospitalization, extended physical therapy or
rehabilitation (for those patients that are capable of entering
such programs), and many require long-term institutional or family
care.
About MultiStem®
MultiStem® cell therapy (invimestrocel) is a patented
regenerative medicine product candidate in clinical development
that has shown the ability to promote tissue repair and healing in
a variety of ways, such as through the production of therapeutic
factors in response to signals of inflammation and tissue damage.
MultiStem therapy’s potential for multidimensional therapeutic
impact may distinguish it from traditional biopharmaceutical
therapies focused on a single mechanism of benefit. MultiStem
represents a unique "off-the-shelf" stem cell product candidate
that can be manufactured in a scalable manner, may be stored for
years in frozen form, and is administered without tissue matching
or the need for immune suppression. Based upon favorable outcome
data, its novel mechanisms of action, and favorable and consistent
tolerability data in clinical studies, we believe that MultiStem
therapy may provide a meaningful benefit to patients, including
those suffering from serious diseases and conditions with unmet
medical need.
About Athersys
Athersys is a biotechnology company engaged in the discovery and
development of therapeutic product candidates designed to extend
and enhance the quality of human life. The Company is developing
its MultiStem® cell therapy product, a patented, adult-derived
"off-the-shelf" stem cell product, initially for disease
indications in the neurological, inflammatory and immune,
cardiovascular, and other critical care indications and has several
ongoing clinical trials evaluating this potential regenerative
medicine product. Athersys has forged strategic partnerships and a
broad network of collaborations to further advance MultiStem cell
therapy toward commercialization. Investors and others should note
that we may post information about the Company on our website at
www.athersys.com and/or on our accounts on Twitter, Facebook,
LinkedIn or other social media platforms. It is possible that the
postings could include information deemed to be material
information. Therefore, we encourage investors, the media and
others interested in the Company to review the information we post
on our website at www.athersys.com and on our social media
accounts. Follow Athersys on Twitter at www.twitter.com/athersys.
Information that we may post about the Company on our website
and/or on our accounts on Twitter, Facebook, LinkedIn or other
social media platforms may contain forward-looking statements
within the meaning of the Private Securities Litigation Reform Act
of 1995 that involve risks and uncertainties. You should not place
undue reliance on forward-looking statements contained on our
website and/or on our accounts on Twitter, Facebook, LinkedIn or
other social media platforms, and we undertake no obligation to
publicly update forward-looking statements, whether as a result of
new information, future events or otherwise.
About Healios
Healios is Japan’s leading clinical stage biotechnology company
harnessing the potential of stem cells for regenerative medicine.
It aims to offer new therapies for patients suffering from diseases
without effective treatment options. Healios is a pioneer in the
development of regenerative medicines in Japan, where it has
established a proprietary, gene-edited “universal donor” induced
pluripotent stem cell (iPSC) line to develop next generation
regenerative treatments in immuno-oncology, ophthalmology, liver
diseases, and other areas of severe unmet medical need. Healios’
lead iPSC-derived cell therapy candidate, HLCN061, is a next
generation NK cell treatment for solid tumors that has been
functionally enhanced through gene-editing. Its near-term pipeline
includes the somatic stem cell product HLCM051 (MultiStem®), which
is currently being evaluated in Japan in Phase 2/3 and Phase 2
trials in ischemic stroke and acute respiratory distress syndrome
(ARDS), respectively. Healios was established in 2011 and has been
listed on the Tokyo Stock Exchange since 2015 (TSE Growth: 4593).
https://www.healios.co.jp/en.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of 1995
that involve risks and uncertainties. These forward-looking
statements relate to, among other things, statements regarding the
potential benefits of our MultiStem product candidate; anticipated
results of clinical trials involving our MultiStem product
candidate; the expected timetable for development of our product
candidates, our growth strategy, and our future financial
performance, including our operations, economic performance,
financial condition, prospects, and other future events. We have
attempted to identify forward-looking statements by using such
words as “anticipates,” “believes,” “can,” “continue,” “could,”
“estimates,” “expects,” “intends,” “may,” “plans,” “potential,”
“should,” “suggest,” “will,” or other similar expressions. These
forward-looking statements are only predictions and are largely
based on our current expectations. A number of known and unknown
risks, uncertainties, and other factors could affect the accuracy
of these statements. Some of the more significant known risks that
we face are the risks and uncertainties inherent in the process of
discovering, developing, and commercializing products that are safe
and effective for use as therapeutics, including the uncertainty
regarding market acceptance of our product candidates and our
ability to generate revenues. The following risks and uncertainties
may cause our actual results, levels of activity, performance, or
achievements to differ materially from any future results, levels
of activity, performance, or achievements expressed or implied by
these forward-looking statements: the possibility of unfavorable
results from ongoing and additional clinical trials involving
MultiStem; the risk that positive results in a clinical trial may
not be replicated in subsequent or confirmatory trials or success
in an early stage clinical trial may not be predictive of results
in later stage or large scale clinical trials; our ability to raise
capital to fund our operations, including but not limited to, our
ability to access our traditional financing sources and to continue
as a going concern; our ability to regain compliance with the
requirement to maintain a minimum closing bid price of $1.00 per
share as set forth in Nasdaq Listing Rule 5550(a)(2); the timing
and nature of results from MultiStem clinical trials, including the
MASTERS-2 Phase 3 clinical trial evaluating the administration of
MultiStem for the treatment of ischemic stroke; the success of our
MACOVIA clinical trial evaluating the administration of MultiStem
for the treatment of COVID-19 induced ARDS, and the MATRICS-1
clinical trial being conducted with The University of Texas Health
Science Center at Houston evaluating the treatment of patients with
serious traumatic injuries; the possibility that the COVID-19
pandemic could continue to delay clinical site initiation, clinical
trial enrollment, regulatory review and potential receipt of
regulatory approvals, payments of milestones under our license
agreements and commercialization of one or more of our product
candidates, if approved; the availability of product sufficient to
meet commercial demand shortly following any approval, such as in
the case of accelerated approval for the treatment of COVID-19
induced ARDS; the impact on our business, results of operations and
financial condition from the ongoing and global COVID-19 pandemic,
or any other pandemic, epidemic or outbreak of infectious disease
in the United States; the possibility of delays in, adverse results
of, and excessive costs of the development process; our ability to
successfully initiate and complete clinical trials of our product
candidates; the impact of the COVID-19 pandemic on the production
capabilities of our contract manufacturing partners and our
MultiStem trial supply chain; the possibility of delays, work
stoppages or interruptions in manufacturing by third parties or us,
such as due to material supply constraints, contamination,
operational restrictions due to COVID-19 or other public health
emergencies, labor constraints, regulatory issues or other factors
which could negatively impact our trials and the trials of our
collaborators; uncertainty regarding market acceptance of our
product candidates and our ability to generate revenues, including
MultiStem cell therapy for neurological, inflammatory and immune,
cardiovascular and other critical care indications; changes in
external market factors; changes in our industry’s overall
performance; changes in our business strategy; our ability to
protect and defend our intellectual property and related business
operations, including the successful prosecution of our patent
applications and enforcement of our patent rights, and operate our
business in an environment of rapid technology and intellectual
property development; our possible inability to realize
commercially valuable discoveries in our collaborations with
pharmaceutical and other biotechnology companies; our ability to
meet milestones and earn royalties under our collaboration
agreements, including the success of our collaboration with
Healios; our collaborators’ ability to continue to fulfill their
obligations under the terms of our collaboration agreements and
generate sales related to our technologies; the success of our
efforts to enter into new strategic partnerships and advance our
programs; our possible inability to execute our strategy due to
changes in our industry or the economy generally; changes in
productivity and reliability of suppliers; the success of our
competitors and the emergence of new competitors; and the risks
mentioned elsewhere in our Annual Report on Form 10-K for the year
ended December 31, 2021 under Item 1A, “Risk Factors” and our other
filings with the SEC. You should not place undue reliance on
forward-looking statements contained on our website and/or on our
accounts on Twitter, Facebook, LinkedIn or other social media
platforms, and we undertake no obligation to publicly update
forward-looking statements, whether as a result of new information,
future events or otherwise.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220520005089/en/
Ivor Macleod Chief Financial Officer Tel: (216) 431-9900
ir@athersys.com
Karen Hunady Director of Corporate Communications and Investor
Relations Tel: (216) 431-9900 khunady@athersys.com
David Schull Russo Partners, LLC Tel: (212) 845-4271 or (858)
717-2310 David.schull@russopartnersllc.com
Peter Vozzo ICR Westwicke Tel: (443) 213-0505
peter.vozzo@westwicke.com
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